E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Catecholaminergic polymorphic ventricular tachycardia |
Katekoliamiiniherkkä monimuotoinen kammiotiheälyöntisyys |
|
E.1.1.1 | Medical condition in easily understood language |
Catecholaminergic polymorphic ventricular tachycardia |
Katekoliamiiniherkkä monimuotoinen kammiotiheälyöntisyys |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Cardiovascular Diseases [C14] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10036095 |
E.1.2 | Term | Polymorphic ventricular tachycardia |
E.1.2 | System Organ Class | 100000004849 |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Assess the antiarrhythmic effects of dantrolene |
Dantroleenin rytmihäiriöitä estävän vaikutuksen tutkiminen |
|
E.2.2 | Secondary objectives of the trial |
Assess the effects of dantrolene on cardiac repolarization |
Dantroleenin kammiorepolarisaatioon kohdistuvien vaikutusten tutkiminen |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Exercise-provoked premature ventricular complexes
RYR2 mutation
Age>17
On-going treatment with betablockers
|
RYR2-geenin mutaatiosta johtuva, rasituksessa ilmaantuva kammiolisälyöntisyys
Vähintään 18 vuoden ikä
Beetasalpaajalääkitys käytössä |
|
E.4 | Principal exclusion criteria |
calcium-channel blocker or metoclopramide medication,
inability to do exercise-stress test with bicycle,
systolic blood pressure over 160 or diastolic over 95 mmHg,
clinically significant angina pectoris or valvular disease,
congenital heart disease
At resting ECG PQ interval >200 ms, QRS>120 ms, LVH, pre-excitation
|
kalsiumkanavasalpaaja- tai metoklopramidilääkitys
- raskaus
- liikuntaa rajoittava tuki- ja liikuntaelimistön sairaus
- verenpaine levossa yli 160 (S) tai yli 95 (D)
- aiemmin todettu kliinisesti merkittävä sepelvaltimotauti tai läppävika, synnynnäinen sydänvika
EKG: PQ>200 ms, QRS>120 ms, LVH, delta-aalto
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
at each minute of exercise: heart rate, number of premature ventricular complexes, number of longest ventricular salvo during exercise stress test after study drug infusion |
- sinustaajuus levossa
- kammiolisälyöntien määrä levossa 5 minuutin aikana
-syketaso, jossa kammiolisälyönnit ilmaantuvat ensi kertaa rasituksen aikana
-kammiolisälyöntien määrä/minuutti kunkin rasitusportaan aikana
-pisimmän perättäisten kammiokompleksien sarjan pituus |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
at each minute of exericse |
kunkin rasitusportaan (á 1 min) aikana
|
|
E.5.2 | Secondary end point(s) |
duration of the exercise in minutes
sinus rate at the end of each minute
achieved maximum sinus rate |
- rasituksen kesto minuuteissa
- sinustaajuus kunkin kuormaportaan päättyessä
- saavutettu maksimaalinen sinustaajuus
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
First eight minutes of the recovery phase |
palautumisvaiheen 8 ensimmäistä minuuttia |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.3.1 | Comparator description |
rasituskoe pelkän beetasalpaajan aikana ilman dantroleenia (keittosuolainfuusio) |
exercise stress test with betablocker only (NaCl 0.9 infusion) |
|
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
Three completed exericse stress tests |
Kolme suoritettua rasituskoetta |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |